This is the first FDA approved medication for the rare and progressive neurodegenerative disease, Niemann-Pick Disease Type C ...

New anecdotal evidence suggests some chemoimmunotherapy treatments may improve pulmonary arterial hypertension symptoms, but the overall evidence remains murky.

GeneCQ uses integrated medical and pharmacy claims data to provide insight into the exposure to the costs for gene therapies.

Results looking at the first season of RSV use shows that it is 75% effective at preventing RSV-associated hospitalizations ...